New research published in the issue of the journal The Lancet Haematology has revealed that 88.2% of patients receiving pharmacoscopy-guided treatment achieved partial or complete remission compared to 23.5% to their own previous treatment. Further, the median progression-free survival increased four-fold.

Retrospectively, pharmacoscopy also predicted the response of AML patients to first-line treatment with 90% accuracy. The results showed that pharmacoscopy could assist decision-making of the responsible clinicians effectively and thus represent a powerful tool for practical precise and personalized medicine.

Patients suffering from refractory and relapsed blood cancers often have few treatment options and short survival times. At this stage, identifying effective therapies can be challenging for doctors. Even advanced genetic analyses, due to the high heterogeneity of cancer cells and the impact of the various mutations on their drug response, do often not suffice to instruct personalized treatments.

Pharmacoscopy, a technology developed by scientists and tested for its clinical efficacy by clinicians, offers a functional approach: hundreds of drug options can be quickly pre-tested ex vivo in small liquid biopsy samples collected from individual patients. The effects of each drug on the individual cells are quantified using high-throughput and high-content automated confocal microscopy.

In combination with specially developed analysis methods and machine learning and other unique algorithms, pharmacoscopy allows quantification of never-before visualized phenotypes. While the clinical study is still recruiting, an interim analysis of the program showed that 88.2% of the patients recruited who received pharmacoscopy-monitored personalized therapies achieved partial or complete remission, while only 23.5% responded similarly well to their previous respective treatments.

In addition, the median progression-free survival of patients who were treated in accordance with pharmacoscopy-guided therapy increased from 5.7 week to 22.6 weeks compared to their last line of treatment. Further, in a retrospective study organized to specifically determine the ability of the method to stratify responding and non-responding newly-diagnosed patients with acute myeloid leukemia (AML), resulted in 90% accuracy.

Before, such accuracy in prediction of treatment outcome was unachievable, with or without genetic assays. Having a robust, fast, and reliable predictive test at disposal during the patient treatment process, especially at the time of relapse where a new intervention must be selected quickly, will change how medical doctors prioritize drugs to use for late-stage patients.

Evidence that the pharmacoscopy approach is helpful for clinical evaluation of therapy is wonderful. Single-cell functional analysis of primary material gives unprecedented resolution and precision that we are sure to further develop in the future to address yet more diseases Giulio Superti-Furga, principal investigator of the clinical trial concluded.